Amicus Therapeutics Receives Breakthrough Therapy Designation for Pompe Treatment
Amicus Therapeutics announced today that they have received Breakthrough Therapy Designation (BTD) for their AT-GAA treatment for Pompe disease.
Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Study of AT-GAA in Patients with Pompe Disease
Amicus Therapeutics has announced that the first patient has been dosed in their Phase 3 study of AT-GAA for patients with Pompe disease.
Valerion Initiates VAL-1221 Dosing in Patients with Pompe Disease
CONCORD, Mass., July 11, 2017 /PRNewswire/ — Valerion Therapeutics, a clinical-stage biotechnology company that specializes in the development of therapies for orphan genetic diseases, today announced that it has initiated dosing in a Phase 1/2 clinical trial evaluating VAL-1221 in patients with late-onset Pompe disease.
Amicus Therapeutics Announces Positive Functional Data from Initial Patients in Pompe Phase 1/2 Study
CRANBURY, N.J., (May 15, 2017) (GLOBE NEWSWIRE) – Amicus Therapeutics (Nasdaq:FOLD) today announced positive functional data from initial patients in a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221 in patients with Pompe disease. Patients who completed six months of treatment with ATB200/AT2221 showed improvements in the six-minute walk test (6MWT) distance and other measures of motor function, in addition to stability or improvements in forced vital capacity (FVC).
Valerion Reports Preclinical Potential for Pompe Treatment
CONCORD, Mass. – (April, 2017) – Valerion Therapeutics, part of the Alopexx Enterprises portfolio of companies, has announced the development of VAL-1221, a fusion protein that combines its technology with recombinant human acid alpha-glucosidase (rhGAA) to improve delivery of rhGAA to affected tissues in patients with Pompe disease.
Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease
Concord, MA – (March 3, 2017) – Valerion Therapeutics announced today that it has developed a fusion protein, VAL-1221, which combines its proprietary antibody delivery technology with recombinant human acid alpha-glucosidase (rhGAA) to improve the delivery of rhGAA into affected tissues of patients with Pompe disease (Glycogen Storage Disease, Type II; GSDII).
Sanofi Genzyme Includes Vulnerable Kiwis in Their Compassionate Access Program
Masterton, New Zealand – (February 25, 2017) – The New Zealand Pompe Network is ecstatic to announce that Sanofi Genzyme has offered Pompe disease patients in New Zealand access to their International Compassionate Access Program (ICAP) for the drug Myozyme®. A huge THANK YOU to Sanofi Genzyme for their generosity and their humanitarian program.
Amicus Therapeutics Presents Important New Scientific Findings and Preclinical Data for Pompe Program at WORLDSymposium™ 2017
CRANBURY, N.J. and SAN DIEGO, Feb. 15, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today presented new scientific findings and preclinical data on functional outcomes in an oral presentation and poster1 at the 13thAnnual WORLDSymposium™ in San Diego, CA. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake in muscles, co-administered with AT2221, a pharmacological chaperone designed to stabilize ERT in circulation.
Amicus Therapeutics Presents Additional Positive Preliminary Phase 1/2 Data at WORLDSymposium™ 2017
CRANBURY, N.J. and SAN DIEGO, Feb. 15, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today presented additional positive preliminary data from a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221 in a poster1 at the 13th Annual WORLDSymposium™ in San Diego, CA. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone.
Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the first patient has been enrolled and received an infusion in a pivotal Phase 3 clinical trial named COMET for the investigational therapy neoGAA.